TRIM-EDICINE INC — Department of Health and Human Services SBIR Phase II: 400

Description

Project Summary This direct Phase II SBIR proposal focuses on the development of recombinant human MGrhMGa novel tissue repair proteinas a therapeutic candidate targeting injury of renal epithelium to protect against acute kidney injuryAKIAKI is commonly encountered in the hospital and outpatient settings and is associated with a high rate of mortalityCurrentlythere is no effective means for preventing or treating AKIMGis a member of the TRIM family proteins that participates in repair of injury to the cell membraneand a vital component for reno protection under both physiologic and pathophysiologic settingsResearch and development effort at TRIM edicinea university spin off biotechnology companyhas established the following proof of concept data supporting rhMGas a potential therapeutic reagent for AKIFirstthe chemistrymanufacture and controlCMCprocess for rhMGhas been established that allows for scale up production of rhMGto support our pre clinical and future clinical studiesSecondpreliminary toxicological studies in rodent and dog models support the safety for systemic application of rhMGThirdtransgenic mice with sustained elevation of MGin the bloodstream are healthy with enhanced lifespan and display remarkable tissue healing capacityFourthpilot study with a canine model of AKI shows that prophylactic administration of rhMGcan preserve kidney function following ischemia reperfusion injuryStudies proposed in this SBIR project involve joint development efforts between TRIM edicine and The Ohio State Universityaiming to accomplish the IND enabling studies for developing rhMGas an injectable therapeutics for AKI treatmentFive milestones with deliverable criteria are proposed in this applicationto complete the CMC for producing sufficient rhMGprotein to support pre clinicaltoxicological and human clinical trialsMilestoneto develop assays for quality control of rhMGMilestoneto establish the pharmacodynamic property of rhMGin the canine model of AKIMilestoneto conduct safety and toxicological evaluation of rhMGper FDA guidanceMilestoneand to assemble IND for rhMGin AKI treatmentMilestoneCompletion of these milestones will enable us to file an IND application to the FDA for initiating a Phaseclinical trial for conducting anascending dose study of the safety of a single intravenous infusion of rhMGin human subjects with stable ischemic heart diseases who are at risk of developing AKI during cardiothoracic surgery